Roche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)
Collaboration offers new hope for a potential treatment for the leading genetic cause of death in infants and toddlers
BASEL, Switzerland, SOUTH PLAINFIELD, N.J., and NEW YORK,
November 29, 2011, 2011 /PRNewswire/ --Roche (SIX: RO, ROG; OTCQX:
RHHBY), PTC Therapeutics, Inc. (PTC) and the SMA Foundation,
announced today a licensing agreement for PTC's Spinal Muscular
Atrophy (SMA) programme. SMA is a genetic neuromuscular disorder
that causes muscle weakness. One in every 10,000 children born is
affected with the disorder, which currently has no effective
treatment.
PTC Therapeutics' programme has been developed in partnership with
the SMA Foundation, which will remain active in the collaboration.
The SMA Foundation was established in 2003 by Loren Eng and Dinakar
Singh to accelerate the development of a treatment for SMA.
SMA is caused by a missing or defective SMN1 gene, which results in
reduced levels of the survival motor neuron (SMN) protein. The
compounds in PTC's research treat the underlying cause of the
disorder and demonstrate increases in SMN levels in nervous system,
muscles and other tissues in SMA models. SMA is a rare disorder and
could be eligible for orphan status by regulatory authorities,
thereby potentially reducing the time needed for a drug to reach
patients.
Under the terms of the agreement, Roche gains an exclusive
worldwide license to PTC's SMA programme, which includes three
compounds currently in preclinical development, as well as
potential back-up compounds. PTC receives USD30 million as an
upfront payment, up to USD460 million upon successful completion of
certain development and commercialization milestones, and up to
double-digit royalties on commercial sales. Development will be
overseen by a joint steering committee comprised of members from
Roche, PTC and the SMA Foundation.
"This collaboration brings us one step closer to developing a
treatment for a condition that has a profound effect on the lives
of many thousands of children and their parents worldwide," said
Dinakar Singh, Chairman of the SMA Foundation. "We are very
optimistic that, by building on the pioneering efforts of PTC
Therapeutics, Roche can help us realise what we have been working
so hard to achieve."
Luca Santarelli, Global Head of Roche Neuroscience, said: "We found
the science behind this programme very compelling, with the
potential to help treat a currently incurable condition. This is
the essence of Roche's entire strategy, focused on solid science
and high unmet clinical need, and these compounds bolster our rich
pipeline in Central Nervous System diseases. As an established
partner of Roche, we already have experience with PTC's scientific
approach. Together with the involvement of the SMA Foundation, we
now have the opportunity to make a significant impact in the
treatment of SMA."
"Having been a partner with Roche for several years, we have every
confidence that the combination of our own expertise and Roche's
considerable capabilities in clinical development, biomarkers and
diagnostics will help us maximize the potential for this
programme," commented Stuart W. Peltz, Ph.D., President and CEO of
PTC. "We are delighted that the SMA Foundation continues to be an
active participant in the collaboration as we share a strong
commitment to advancing this innovative potential treatment as
rapidly as possible."
Roche and PTC announced their first collaboration in September of
2009 for the development of orally bioavailable small molecules
utilizing PTC's technology called Gene Expression Modulation by
Small-molecules (GEMS™). The SMA programme has been developed
by PTC utilizing a different scientific approach than GEMS, called
alternative splicing.
About the SMA Foundation
Founded in 2003, the Spinal Muscular Atrophy Foundation is a
nonprofit organization dedicated to accelerating progress towards a
treatment for Spinal Muscular Atrophy through targeted funding of
clinical research and novel drug development efforts. Since its
inception, the Foundation has awarded over $100 million for SMA
research. In addition, the Foundation is committed to raising
awareness and generating support for increased research efforts in
SMA among the leaders of industry and government. For more
information, visit the SMA Foundation website at www.smafoundation.org.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered
small-molecule drugs that target post-transcriptional control
processes. Post-transcriptional control processes regulate the rate
and timing of protein production and are of central importance to
proper cellular function. PTC's internally discovered pipeline
addresses multiple therapeutic areas, including rare genetic
disorders, oncology and infectious diseases. PTC has developed
proprietary technologies that it applies in its drug discovery
activities and that have served as the basis for collaborations
with leading biopharmaceutical companies such as AstraZeneca,
Celgene, Genzyme, Merck, Pfizer and Roche. For more information,
visit the company's website at www.ptcbio.com.
About Roche
Headquartered in Basel, Switzerland, Roche is a leader in
research-focused healthcare with combined strengths in
pharmaceuticals and diagnostics. Roche is the world’s largest
biotech company with truly differentiated medicines in oncology,
virology, inflammation, metabolism and CNS. Roche is also the world
leader in in-vitro diagnostics, tissue-based cancer diagnostics and
a pioneer in diabetes management. Roche’s personalised
healthcare strategy aims at providing medicines and diagnostic
tools that enable tangible improvements in the health, quality of
life and survival of patients. In 2010, Roche had over 80,000
employees worldwide and invested over 9 billion Swiss francs in
R&D. The Group posted sales of 47.5 billion Swiss francs.
Genentech, United States, is a wholly owned member of the Roche
Group. Roche has a majority stake in Chugai Pharmaceutical, Japan.
For more information: www.roche.com
For more information please contact
Roche
Sharon Valdettaro +41 61 688 9655
Sharon.valdettaro@roche.com
PTC Therapeutics, Inc.
Jane Baj (PTC) +1 (908) 912-9167
jbaj@ptcbio.com
Sheryl Seapy (Pure Communications) +1 (949) 608-0841
sheryl@purecommunicationsinc.com
SMA Foundation
Sergey Paushkin, MD, PhD +1 (646) 253-7100
spaushkin@smafoundation.org
Posted: November 2011
